March 2025

The neuromuscular junction (NMJ) disorders are a group of conditions that disrupt the communication between motor neurons and muscles, resulting in muscle weakness, fatigue, problems with movement and mobility, and, in severe cases, paralysis. Recognized NMJ disorders include the autoimmune disorders myasthenia gravis (MG) and Lambert-Eaton myasthenic syndrome (LEMS), the genetic conditions known as congenital myasthenic…

Know More

Researchers at AskBio are seeking adults with genetically confirmed limb-girdle muscular dystrophy (LGMD) type 2I/R9 to participate in a phase 1/2 clinical trial (LION-CS101) to evaluate the safety and tolerability of the investigational gene therapy AB-1003 to treat LGMD2I/R9. LGMD2I/R9 is a rare form of LGMD caused by changes in the FKRP gene and is associated…

Know More

PPMD is deeply disappointed that Dr. Peter Marks has made the decision to leave his role as Director of the FDA’s Center for Biologics Evaluation and Research (CBER). In his resignation letter, addressed to Acting FDA Commissioner Sarah Brenner, Dr. Marks indicated that his departure will be effective April 5, 2025. This leadership change is…

Know More

Adding more movement or physical activity to each day is a common goal among people living with neuromuscular disease. However, muscle fatigue, weakness, and limited strength or range of motion can make increasing physical activity a challenge. The age-old conundrum of “if you don’t use it, you lose it” conflicts with the need for rest…

Know More

Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung function for boys with Duchenne muscular dystrophy (DMD), new analyses indicate. The studies were presented in a series of posters at this year’s meeting of the Muscular Dystrophy Association (MDA). Duvyzat is an oral therapy developed…

Know More

​Wave Life Sciences has announced encouraging interim results from its Phase 2 FORWARD-53 clinical trial of WVE-N531, an exon skipping oligonucleotide being investigated for the treatment of individuals with Duchenne who are amenable to exon 53 skipping. According to Wave, FORWARD-53 achieved all trial goals, demonstrating sustained exon skipping, muscle concentrations, and dystrophin restoration through…

Know More

I was tempted to do a play on the show “America’s Got Talent” and title this column “FSHDers got talent,” but I knew the improper grammar wouldn’t get past my excellent editors. That being said, after participating in a recent FSHD Society wellness group session, I know that my fellow FSHDers — those of us…

Know More

A team has discovered a possible therapeutic approach to repair injured muscles either from aging or degenerative muscle disorders.

Know More

Patient advocate Donavon Decker has been given the 2025 MDA Legacy Award for Community Impact in Research, recognizing his efforts across decades on behalf of the muscular dystrophy community. Decker, who has limb-girdle muscular dystrophy (LGMD) type 2D, was honored at the Muscular Dystrophy Association (MDA)’s annual meeting, recently held in Dallas and virtually. “It’s always…

Know More

REGENXBIO Inc. has reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product delivering a micro-dystrophin transgene via AAV8 for individuals with Duchenne. According to REGENXBIO, the new data from the age 1-3 cohort builds on the favorable safety…

Know More